Research Roundup: FDA Commissioner to step down, a Grand Challenge for neglected diseases, funding for reproductive health research and development, and more

In this guest post, Isabela Ribeiro, MD—head of the Chagas program at the Drugs for Neglected Diseases initiative (DNDi)—discusses the devastating toll of Chagas and ongoing efforts to develop new treatments and drug regimens for the disease. Valentine’s Day seems like a fitting occasion to shine some needed light on a disease that can begin with a late-night “kiss” but ends with a broken heart. I’m talking about Chagas, the leading cause of infectious heart disease in Latin America. The T. cruzi parasite that causes the illness is transmitted by the “kissing bug,” a blood-sucking insect found in the Americas that thrives in houses made from straw, sticks, and adobe brick. Kissing bugs feed at night, biting the face, sometimes around the lips (hence their name), where they can deposit feces infected by the T. cruzi parasite. Chagas disease can also be transmitted from mother to child or even through blood transfusions. The acute stage of Chagas disease happens soon after infection. But the chronic stage lasts a lifetime as the parasites hide in the heart and other organs. More than 30 percent of those infected will develop serious cardiac, digestive, or neurological disorders. Up to eight million people are infected and more than 12,000 deaths occur each year, in most cases from heart failure. What is so remarkable about this silent killer is the deep neglect that surrounds it. Chagas is endemic to all countries in Latin America and increased migration and population movement means there are now cases throughout the world, including approximately 300,000 in the United States. Less than 1 percent of those infected around the globe are estimated to be on treatment. Only two drugs exist to treat Chagas disease—benznidazole and nifurtimox. Both are effective, but have undesirable side effects and require a long treatment course. Nevertheless, governments can and must do much more to scale up access to testing and treatment with these existing tools. In the United States, neither drug has been approved by the US Food and Drug Administration. Treatments are only available through the US Centers for Disease Control and Prevention under investigational protocols and compassionate use. The past decade has seen new actors enter into Chagas research and development (R&D), including DNDi, the Sabin Vaccine Institute, and other institutions. New technologies are enabling researchers to screen much larger libraries of compounds for potential drug candidates. The first clinical trials for Chagas in more than 40 years are being held, providing both new formulations and new regimens of existing drugs and desperately-needed knowledge and data about this poorly understood disease. Drug discovery is a slow process with many ups and downs. One class of antifungals known as “azoles” generated a good deal of hope, but recent clinical trials have shown that they are not as effective as current drugs. Nevertheless, DNDi is currently looking into combination treatments with these drugs. Increased open collaboration should hopefully lead to new drug candidates. One important development is DNDi’s Lead Optimization Latin America project, which is a rare example of early R&D for neglected diseases taking place in an “endemic” region. There is still a long way to go to break the silence around this disease and ensure the needs of millions of neglected patients are met. As is often the case with neglected diseases, it is the poorest and most vulnerable communities that are the most affected. New technologies to test, treat, and prevent the parasite hold the key to future progress against Chagas disease, but a larger global effort is needed today to scale up access to treatment for those who need it most.

WHO is moving forward on creating of new pooled fund to finance R&D. PATH/Patrick McKern In this guest post, Alexandra Heumber—head of policy affairs at the Drugs for Neglected Diseases initiative—discusses actions taken during the World Health Organization (WHO)’s Executive Board meeting which could impact financing for the research and development (R&D) of new health technologies for developing countries. Earlier this week, the WHO’s Executive Board concluded its 136th annual executive board meeting. Given the Ebola epidemic and ongoing debates about chronic underinvestment in health R&D for developing countries, it was no surprise that global health R&D featured prominently on the board’s agenda. The board discussed the follow-up of the report of the Consultative Expert Working Group on Research and Development (CEWG): Financing and Coordination, which proposed establishing a voluntary pooled fund to finance global health R&D. The board agreed to move forward with establishing this fund at the WHO Special Programme for Research and Training in Tropical Diseases (TDR). The fund will finance R&D for type II and III diseases, as well as specific needs of developing countries in relations to type I diseases. During discussions, member states stressed the importance of this fund, recalling the CEWG recommendations and citing the Ebola crisis and growing antimicrobial drug resistance in justifying its urgent need. At the same time, member states recognized that the success of the fund will depend on securing adequate funding, particularly new funding from new donors. There was broad agreement among member states representing different regions to move forward in creating the fund and more broadly in setting up the four elements to serve as a framework to monitor, coordinate, and finance global health R&D based on the CEWG report. These include: The global R&D observatory to monitor funding for R&D and expenditures A coordinating body with a scientific review committee The pooled fund at TDR Ongoing demonstration projects to illustrate how the fund would work and how CEWG principles such as delinkage—separating drug prices from the cost of R&D—and incentive mechanisms could be implemented During the board meeting, several member states pledged funding to advance these ongoing efforts: Switzerland pledged US$6 million to the demonstration projects and observatory budget line, of which $4 million has already been allocated to the TDR pooled fund. The remaining $2 million will be awarded through matching grants from low- and middle-income countries (LMICs) at the rate of $1 for every $2 contributed. Forty-thousand will be used to start the global health R&D observatory. Norway pledged $1.3 million including$650,000 to the TDR pooled fund and $650,000 to be awarded through matching grants from LMICs, again at the rate of $1 for every $2 contributed. Brazil pledged $1 million. It is encouraging to see the overall CEWG process being reinvigorated and concrete outcomes moving forward. Nevertheless, more mobilization by stakeholders and member states, including political and financial commitments, must occur. Public leadership by all countries is now crucial to make the proposed framework a reality.